Alzheimer’s Disease (AD) is a debilitating, progressive neurodegenerative disorder that impacts over 6 million people in the United States alone. AD is characterized primarily by severe dementia. There is no cure available for AD, and current treatments do not target the root cause of AD. However, the FDA recently approved Aducanumab (under the name Aduhelm) as the first drug to attempt to stop the progression of AD by directly targeting its pathology.
AD is a disorder that affects the brain tissue, meaning the disease is caused by some pathological change in the brain. While scientists may be familiar with the symptomatology of AD, the only way AD can be definitively diagnosed is upon examination of the brain post mortem. One of the hallmark pathologies of AD is the presence of neurofibrillary tangles (NFTs), which are abnormal accumulations of the protein tau. Tau is found in brain cells and plays an important structural role for the cell. NFT formation contributes to the brain damage that leads to symptoms of AD. Another major pathology in AD is Aβ (amyloid-beta) plaque formation. Aβ proteins are a class of proteins naturally found in brain tissue, however they form characteristic plaques in AD; these plaques have been a major point of study and are the target of Aducanumab.
In Alzheimer’s Disease, proteins accumulate to form aggregates in an abnormal manner.
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Specifically, Aducanumab is an antibody that targets aggregates of Aβ, which, as discussed above, are found in AD. Aducanumab has been vastly studied in animal models and more recently, in the clinic. PET scans of AD patients can identify levels of Aβ and, in AD patients treated with Aducanumab, have shown reduced levels of Aβ . There have also been identifiable, albeit smaller, reductions in Tau-protein and NFT levels. Scientists believe this is due to Aβ and Tau-protein interactions in AD pathology. Additionally, the drug has been shown to, in clinical trials, provide a cognitive benefit for AD patients. These findings together contributed to the FDA approval of the drug, making history and paving the way for future research investigating treatments for AD.
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